我国小核酸药物研发与注册的相关问题研究-中国药房.PDF

我国小核酸药物研发与注册的相关问题研究 ＊ # 董 浩 ，梁 毅（中国药科大学国际医药商学院，南京 210009） 中图分类号 R95 文献标志码 A 文章编号 1001-0408（2016）07-0876-03 DOI 10.6039/j.issn. 1001-0408.2016.07.04 摘 要 目的：为我国小核酸药物（SiRNA）研发与注册提供参考。方法：介绍国内外SiRNA 的研发现状，归纳我国SiRNA 在药品 审评、审批中遇到的问题并提出建议。结果与结论：我国SiRNA研发与国外发达国家相比至少有5～10年的差距。SiRNA 注册类 别不明确，申请者缺乏指导性文件，缺乏符合条件的生产车间，药品注册部门审批、审批时效性较差。建议药品注册部门开展SiR- NA 注册改革试点工作，出台SiRNA 注册指南，逐步放宽临床申请条件，鼓励申请者参与国际多中心临床试验，以提高SiRNA 审 评、审批效率。 关键词 小核酸药物；研发；新药；药品注册 Research on the Related Problems of Small Interfering Ribonucleic Acid RD and Registration in China DONG Hao ，LIANG Yi （School of International Pharmaceutical Business ，China Pharmaceutical University ，Nan- jing 210009，China ） ABSTRACT OBJECTIVE ：To provide reference for small interfering ribonucleic acid （SiRNA）RD and registration in China. METHODS ：The situation of SiRNA RD at home and abroad were introduced ，and problems encountered in the evaluation and approval process were summed up to put forward suggestions. RESULTS CONCLUSIONS ：SiRNA has developed slowly in Chi- na ，with a gap of 5-10years at least compared with foreign developed countries. The registration category of SiRNA is not clear ； applicants lack of guidance documents ；production workshop which meet requirement is in shortage ；the efficiency of drug registra- tion allows of no optimism. It is recommended that pharmaceutical registration departments carry out the pilot reform for SiRNA registration ，publish SiRNA registration guide ，relax regulations for IND gradually ，encourage applicants to participate in the inter- national multicenter clinical trials in order to improve the efficiency of SiRNA evaluation and approval efficiency. KEYWORDS Small interfering ribonucleic acid ；RD ；New drug ；Drug registration 小核酸药物（SiRNA）是人工合成的双链核糖核酸，一般由 1.1 国外SiRNA 的研发概况 19～25对核苷酸构成，其主要基于“基因沉默”技术，干扰目标